Introduction to Epidemiology and Study Designs Adapted from

Introduction to Epidemiology and Study Designs Adapted from original PPT by Thomas Songer, Ph. D. – University of Pittsburgh and the Supercourse Team.

What is Epidemiology? • Epidemiology may be regarded simply as the study of disease and health in human populations. • Here disease may be any adverse health outcome such as pre-term birth, it may not necessarily be a disease such as cancer.

Goals of Epidemiological Research 1) Describe the health status of populations by enumerating the occurrence of diseases, obtaining relative frequencies within groups and discovering important trends. 2) Explain the etiology of diseases by determining factors that “cause” specific diseases or trends.

Goals of Epidemiological Research 3) Predict the number of disease occurrences and the distribution of health status within populations. 4) Control the distributions of disease in the population by prevention of new occurrences, eradication of existing cases, prolongation of life for those with disease, or otherwise improving the health status of afflicted persons.

Components of Epidemiology • Measure disease frequency – Quantify disease • Assess distribution of disease – Who is getting disease? – Where is disease occurring? – When is disease occurring? èFormulation of hypotheses concerning causal and preventive factors • Identify determinants of disease – Hypotheses are tested using epidemiologic studies

Types of Primary Studies • Descriptive Studies – describe occurrence of an outcome • Analytic Studies – describe the potential association between exposure and outcome

Basic Question in Analytic Epidemiology • Are exposure and disease linked? Exposure Disease

Basic Questions in Analytic Epidemiology • Look to link exposure and disease – What is the exposure? – Who are the exposed? – What are the potential health effects? – What approach will you take to study the relationship between exposure and effect?

Basic Study Designs and their Application to Epidemiology

Big Picture • To prevent and control disease • In a coordinated plan, look to – identify hypotheses on what is related to disease and may be “causing” it – formally test these hypotheses • Study designs direct how the investigation is conducted

What designs exist to identify and investigate factors in disease?

Study Designs Descriptive Case report Case series Descriptive Epidemiology Analytic RCT Cohort study Case-Control study Case-Crossover study Cross-sectional study Before-After study Ecologic study

Timeframe of Studies • Prospective Study - looks forward, looks to the future, examines future events, follows a condition, concern or disease into the future (looking forward) Study begins here time

Timeframe of Studies • Retrospective Study - “to look back”, looks back in time to study events that have already occurred (looking back) time Study begins here e. g. Case-Control Study

Study Design Sequence Hypothesis formation Case reports Case series Analytic epidemiology Clinical trials Descriptive epidemiology Animal study Hypothesis testing Cohort Casecontrol Crosssectional Lab study

Increasing Knowledge of Disease/Exposure Descriptive Studies Develop hypothesis Case-control Studies Investigate it’s relationship to outcomes Cohort Studies Define it’s meaning with exposures Clinical trials Test link experimentally

Descriptive Studies

Case Reports • Detailed presentation of a single case or handful of cases • Generally report a new or unique finding • e. g. previous undescribed disease • e. g. unexpected link between diseases • e. g. unexpected new therapeutic effect • e. g. adverse events

Case Series • Experience of a group of patients with a similar diagnosis • Assesses prevalent disease • Cases may be identified from a single or multiple sources • Generally report on new/unique condition • May be only realistic design for rare disorders

Case Series Case series From Wikipedia, the free encyclopedia http: //en. wikipedia. org/wiki/Case_series • Advantages • Useful for hypothesis generation • Informative for very rare disease with few established risk factors • Characterizes averages for disorder • Disadvantages • Cannot study cause and effect relationships • Cannot assess disease frequency

Descriptive Studies Case Report One case of unusual findings Case Series Multiple cases of findings Descriptive Epidemiology Study Population-based cases with denominator

Analytical Studies

Study Designs Analytic Epidemiology • Experimental Studies – Randomized Controlled Clinical Trials (RCT) – Community trials • Observational Studies – Group data (i. e. we don’t have subject level info) • Ecologic – Individual data • Cross-sectional • Cohort • Case-control • Case-crossover An Introduction to Epidemiology (CDC) http: //www. cdc. gov/excite/classroom/intro_epi. htm

Experimental Studies • Treatment and/or exposures occur in a “controlled” environment • Planned research designs • Clinical trials are the most well known experimental design. Clinical trials use randomly assigned data. • Community trials use nonrandom data

Observational Studies • Non-experimental • Observational because there is no individual intervention • Treatment and/or exposures occur in a “non-controlled” environment • Individuals can be observed prospectively, retrospectively, or currently (i. e. cross-sectional)

Cross-sectional studies • An “observational” design that surveys exposures and disease status at a single point in time (a cross-section of the population) time Study only exists at this point in time

Observational Studies and Timeframe

Cross-sectional Design factor present No Disease Study population Disease factor absent factor present factor absent time Study only exists at this point in time

Cross-sectional Studies • Often used to study conditions that are relatively frequent with long duration of expression (nonfatal, chronic conditions) • It measures prevalence, not incidence of disease • Example: community surveys • Not suitable for studying rare or highly fatal diseases or a disease with short duration of expression

Cross-sectional studies • Disadvantages • Weakest observational design, (it measures prevalence, not incidence of disease). Prevalent cases are survivors • The temporal sequence of exposure and effect may be difficult or impossible to determine • Usually don’t know when disease occurred • Rare events a problem. Quickly emerging diseases are also problem.

Epidemiologic Study Designs • Case-Control Studies – an “observational” design comparing exposures in disease cases vs. healthy controls from same population – exposure data collected retrospectively – most feasible design where disease outcomes are rare

Case-Control Studies Cases: Disease Controls: No Disease

Case-Control Design factor present factor absent Cases (disease) Study population Controls (no disease) present past time Study begins here

Case-Control Study • Strengths – Less expensive and time consuming – Efficient for studying rare diseases • Limitations – Inappropriate when disease outcome for a specific exposure is not known at start of study – Exposure measurements taken after disease occurrence – Disease status can influence selection of subjects

Hypothesis Testing: Case-Crossover Studies • Study of event triggers within an individual • Still a Case and Control component, but information on both components will come from the same individual who is ultimately a case. • Case component = hazard period which is the time period right before the disease or event onset. For example, period of heavy lifting right before an MI. • Control component = control period which is a specified time interval other than the hazard period. For example, presence or absence of a period of heavy lifting prior to time right before MI.

Epidemiologic Study Designs • Cohort Studies – an “observational” design comparing individuals with a known risk factor or exposure with others without the risk factor or exposure – looking for a difference in the risk (incidence) of a disease over time – best observational design – data usually collected prospectively

Cohort Design Study population free of disease Factor present Factor absent present time Study begins here disease no disease future

Timeframe of Studies • Prospective Study - looks forward, looks to the future, examines future events, follows a condition, concern or disease into the future

Prospective Cohort study Exposed Outcome Non-exposed Outcome Measure exposure and confounder variables Baseline time Study begins here

Timeframe of Studies • Retrospective Study - “to look back”, looks back in time to study events that have already occurred time Study begins here

Retrospective Cohort study Exposed Outcome Non-exposed Outcome Measure exposure and confounder variables Baseline time Study begins here

• Strengths Cohort Study – Exposure status determined before disease detection – Subjects selected before disease detection – Can study several outcomes for each exposure • Limitations – Expensive and time-consuming – Inefficient for rare diseases or diseases with long latency – Loss to follow-up

Experimental Studies • Investigator can “control” the exposure • Akin to laboratory experiments except living populations are the subjects • Generally involves random assignment to groups • Clinical trials are the most well known experimental design • The ultimate step in testing causal hypotheses

Experimental Studies • In an experiment, we are interested in the consequences of some treatment on some outcome. • The subjects in the study who actually receive the treatment of interest are called the treatment group. • The subjects in the study who receive no treatment or a different treatment are called the comparison group or control group.

Epidemiologic Study Designs • Randomized Controlled Trials (RCTs) – a design with subjects randomly assigned to “treatment” and “comparison” groups – provides most convincing evidence of relationship between exposure and effect – not possible to use RCTs to test effects of exposures that are expected to be harmful, for ethical reasons

outcome Experimental Design RANDOMIZATION Intervention Study population Control no outcome baseline future time Study begins here (baseline point)

Epidemiologic Study Designs • Randomized Controlled Trials (RCTs) – the “gold standard” of research designs – provides most convincing evidence of relationship between exposure and effect • trials of hormone replacement therapy in menopausal women found no protection for heart disease, contradicting findings of prior observational studies.

Randomized Controlled Trials • Disadvantages Understanding controlled trials: Why are randomised controlled trials important? by Bonnie Sibbald and Martin Roland http: //www. bmj. com/content/316/7126/201. full – Very expensive – Not appropriate to answer certain types of questions • it may be unethical, for example, to assign persons to certain treatment or comparison groups

Review Questions • What designs can be used to describe the link between exposure and disease? • Describe study design sequence • Describe strengths and weaknesses of each design